(19)
(11) EP 3 585 896 A1

(12)

(43) Date of publication:
01.01.2020 Bulletin 2020/01

(21) Application number: 18711159.6

(22) Date of filing: 14.02.2018
(51) International Patent Classification (IPC): 
C12N 15/113(2010.01)
A61K 48/00(2006.01)
A61K 35/34(2015.01)
 A61P 21/00(2006.01)
A61K 35/28(2015.01)
A61K 35/545(2015.01)
(86) International application number:
PCT/IB2018/050908
(87) International publication number:
WO 2018/154412 (30.08.2018 Gazette 2018/35)
(84) Designated Contracting States:
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
Designated Extension States:
BA ME
Designated Validation States:
MA MD TN

(30) Priority: 22.02.2017 US 201762461840 P

(71) Applicant: CRISPR Therapeutics AG
6300 Zug (CH)

(72) Inventors:
  • LUNDBERG, Ante Sven
    Cambridge Massachusetts 02139 (US)
  • KULKARNI, Samarth
    Cambridge Massachusetts 02139 (US)
  • KLEIN, Lawrence
    Cambridge Massachusetts 02139 (US)
  • PADMANABHAN, Hari Kumar
    Cambridge Massachusetts 02139 (US)

(74) Representative: Vossius & Partner Patentanwälte Rechtsanwälte mbB 
Siebertstrasse 3
81675 München
81675 München (DE)

   


(54) MATERIALS AND METHODS FOR TREATMENT OF MEROSIN-DEFICIENT COGENITAL MUSCULAR DYSTROPHY (MDCMD) AND OTHER LAMININ, ALPHA 2 (LAMA2) GENE RELATED CONDITIONS OR DISORDERS